Ignite Creation Date:
2024-05-05 @ 10:23 PM
Last Modification Date:
2024-10-26 @ 10:18 AM
Study NCT ID:
NCT01096082
Status:
COMPLETED
Last Update Posted:
2013-01-30
First Post:
2010-03-29
Brief Title:
Safety and Efficacy of Lithium Carbonate in Patients With Spinocerebellar Ataxia Type 3
Sponsor:
Hospital de Clinicas de Porto Alegre
Organization:
Hospital de Clinicas de Porto Alegre
Study Overview
Official Title:
Randomized Clinical Trial to Assess the Safety and Efficacy of Lithium Carbonate in Patients With Spinocerebellar Ataxia Type 3
Status:
COMPLETED
Status Verified Date:
2013-01
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Design Phase II-III double-blind parallel placebo controlled randomized Clinical trial
Background Spinocerebellar ataxia type 3 SCA-3 is an autosomal dominant adult-onset neurodegenerative disorder for which there is no current treatment Patients will invariably become dependent from others and unable to walk during the disease course
Hypothesis Lithium Carbonate is safe and effective in treating neurological symptoms and improving quality of life of patients with SCA3
Outcomes
Primary
Phase 2 - To assess safety and tolerability of Lithium Carbonate in patients with SCA3 after 6 months of follow-up
Phase 3 if Phase II study shows safety of therapy - To assess efficacy of Lithium Carbonate in patients with SCA3 through the Neurological Examination Score for SCA 3 NESSCA after 12 months of follow-up
Secondary
1 - To assess efficacy on neurological function ataxic depressive and quality of life scores of Lithium Carbonate in patients with SCA3 through the Scale for the Assessment and Rating of Ataxia SARA 9-Hole Peg Board test 8m walking time PATA repetition rate Click Test SCA Functional Index SCAFI Composite Cerebellar Functional Score CCFS Beck Depression Inventory Barthel Index and WHOQol after 6 and 12 months of follow-up
2 - To assess the effect of Lithium Carbonate in peripheral levels and expression of treatment biomarkers BDNF NSE HDAC GSK-3Beta
Study Duration 12 months
Final analysis of phase 2 safety study at 6 months with continuous monitoring until the end of phase 3 efficacy study
Preliminary analysis of efficacy on ataxia scales at 6 months of study and final analysis of phase 3 at 12 months
Obs A futility analysis will be performed after 12 months of therapy if no statistically significant difference between groups were found This analysis will define if the study will continue until 18 or 24 months of follow-up or will be ended at 12 months
Location Hospital de Clínicas de Porto Alegre
Subjects 60 molecularly diagnosed SCA3 patients from the outpatient unit of the Medical Genetics Service of Hospital de Clínicas de Porto Alegre
Intervention Lithium Carbonate tablets of 300mg Starting dose will be 300mgday with drug titration during 49 days or until achieving the defined target lithium serum level of 05 to 08 mEqL
Background Spinocerebellar ataxia type 3 SCA-3 is an autosomal dominant adult-onset neurodegenerative disorder for which there is no current treatment Patients will invariably become dependent from others and unable to walk during the disease course
Hypothesis Lithium Carbonate is safe and effective in treating neurological symptoms and improving quality of life of patients with SCA3
Outcomes
Primary
Phase 2 - To assess safety and tolerability of Lithium Carbonate in patients with SCA3 after 6 months of follow-up
Phase 3 if Phase II study shows safety of therapy - To assess efficacy of Lithium Carbonate in patients with SCA3 through the Neurological Examination Score for SCA 3 NESSCA after 12 months of follow-up
Secondary
1 - To assess efficacy on neurological function ataxic depressive and quality of life scores of Lithium Carbonate in patients with SCA3 through the Scale for the Assessment and Rating of Ataxia SARA 9-Hole Peg Board test 8m walking time PATA repetition rate Click Test SCA Functional Index SCAFI Composite Cerebellar Functional Score CCFS Beck Depression Inventory Barthel Index and WHOQol after 6 and 12 months of follow-up
2 - To assess the effect of Lithium Carbonate in peripheral levels and expression of treatment biomarkers BDNF NSE HDAC GSK-3Beta
Study Duration 12 months
Final analysis of phase 2 safety study at 6 months with continuous monitoring until the end of phase 3 efficacy study
Preliminary analysis of efficacy on ataxia scales at 6 months of study and final analysis of phase 3 at 12 months
Obs A futility analysis will be performed after 12 months of therapy if no statistically significant difference between groups were found This analysis will define if the study will continue until 18 or 24 months of follow-up or will be ended at 12 months
Location Hospital de Clínicas de Porto Alegre
Subjects 60 molecularly diagnosed SCA3 patients from the outpatient unit of the Medical Genetics Service of Hospital de Clínicas de Porto Alegre
Intervention Lithium Carbonate tablets of 300mg Starting dose will be 300mgday with drug titration during 49 days or until achieving the defined target lithium serum level of 05 to 08 mEqL
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| HCPA FIPE GPPG 09-418 | OTHER_GRANT | HCPA FIPE GPPG 09-418 | None |